FOSTER CITY, Calif. - Mirum Pharmaceuticals, Inc. (NASDAQ: NASDAQ:MIRM) today announced encouraging interim results from two ongoing Phase 2b clinical trials of volixibat, an oral medication being evaluated for the treatment of primary biliary cholangitis (PBC) and primary sclerosing cholangitis (PSC), two rare liver diseases.
The VANTAGE study, which focuses on PBC, reported a statistically significant reduction in pruritus (itchiness) with a placebo-adjusted difference of -2.32 points (p=0.0026) on the Adult ItchRO scale. Moreover, 75% of participants achieved over a 50% decrease in serum bile acids, and improvements in fatigue were observed at week 16 compared to the placebo group.
The VISTAS study on PSC has also passed an efficacy threshold necessary for the continuation of the trial. The independent data review committee has recommended proceeding without modifications to the study, which is evaluating a volixibat dose of 20 mg twice daily.
No new safety concerns were identified during the interim analyses, and the most common side effect was diarrhea, which was mild to moderate in severity. Four serious adverse events were reported, with one occurring in the placebo group. No clinically significant changes in liver biomarkers were observed.
Mirum's Chief Medical Officer, Dr. Joanne Quan, expressed optimism about volixibat's potential as a future treatment option for these conditions, citing the interim results as a promising development for patients struggling with the symptomatic burden of PBC and PSC.
The company is hosting a conference call today to further discuss these interim analyses. The call aims to provide insights into the studies' progress and the potential implications for patients affected by these cholestatic liver diseases.
Volixibat is designed to selectively inhibit the ileal bile acid transporter (IBAT), potentially offering a novel approach to reducing systemic and hepatic bile acids. The drug has been evaluated in more than 400 individuals across various clinical trials.
Mirum Pharmaceuticals focuses on developing treatments for rare diseases and has three approved medications for different liver conditions. The company's pipeline includes volixibat for PBC and PSC, and CHENODAL for cerebrotendinous xanthomatosis, with positive Phase 3 results reported in 2023.
This report is based on a press release statement from Mirum Pharmaceuticals, Inc.
In other recent news, Mirum Pharmaceuticals has been making significant strides in the biopharmaceutical sector. Citi has reaffirmed its Buy rating on Mirum shares, in anticipation of important clinical trial updates for volixibat, a treatment for liver diseases.
The firm is closely monitoring two phase 2b trials, VISTAS and VANTAGE, which are investigating volixibat's efficacy in patients with primary sclerosing cholangitis (PSC) and primary biliary cholangitis (PBC), respectively.
Mirum's other product, LIVMARLI, received a positive opinion from the European Committee for Medicinal Products for Human Use (CHMP) for the treatment of Progressive Familial Intrahepatic Cholestasis (PFIC), a rare genetic liver disease. This recommendation paves the way for potential marketing authorization in the European Union.
Moreover, new data from long-term treatment studies with LIVMARLI will be presented at the upcoming European Association for the Study of the Liver (EASL) Annual Congress, highlighting sustained clinical benefits in patients with Alagille syndrome (ALGS) and PFIC.
In financial developments, Mirum reported a significant increase in financial results for the first quarter of 2024, with net product sales reaching $68.9 million, marking a 137% rise from the prior year. The company forecasts a full-year revenue guidance between $310 million and $320 million. Mirum also reported a solid financial position with $302.8 million in cash and investments. These are the recent developments in Mirum Pharmaceuticals' operations and financial performance.
InvestingPro Insights
Mirum Pharmaceuticals, Inc. (NASDAQ: MIRM) has demonstrated a commitment to addressing rare liver diseases through their promising clinical trials, and the financial metrics from InvestingPro provide a deeper look into the company's current market standing and future potential.
InvestingPro Data for MIRM shows a market capitalization of $1.22 billion, reflecting the market's valuation of the company. Despite the lack of profitability in the last twelve months, as indicated by a negative P/E ratio of -7.7, the company's revenue growth is notably robust. A year-over-year revenue growth of 133.9% signals strong sales performance and potential for future growth.
The company's financial strength is further underscored by a Gross Profit Margin of 73.26%, suggesting that Mirum Pharmaceuticals is efficient in managing its production costs relative to its sales. Additionally, while the company does not pay a dividend, indicating a reinvestment of earnings into further growth and development, an InvestingPro Tip highlights that Mirum's liquid assets exceed its short-term obligations, which is a positive sign for the company's liquidity and financial resilience.
Moreover, the InvestingPro Tips for MIRM include an important consideration for investors: analysts do not expect the company to be profitable this year. This aligns with the interim clinical trial results which, while promising, are yet to translate into approved treatments that could generate revenue.
For readers interested in a more comprehensive analysis, there are additional InvestingPro Tips available, offering detailed insights into Mirum Pharmaceuticals' financial health and market position. To access these tips and make more informed investment decisions, investors can use the coupon code PRONEWS24 to receive an additional 10% off a yearly or biyearly Pro and Pro+ subscription at InvestingPro.
This article was generated with the support of AI and reviewed by an editor. For more information see our T&C.